Conectar
Rare diseases (RDs) affect millions of people worldwide and present major challenges for research due to small patient populations, limited longitudinal data and the difficulty of capturing real-world disease manifestations. Digital technologies provide new opportunities to collect multimodal data directly from patients and their environments. In particular, video recordings, wearable-based physical activity monitoring and digital questionnaires can generate detailed, longitudinal information on symptoms, functional abilities and daily-life impact. The digital tools for rare disorders (DT4RD) protocol aims to develop and evaluate a digital framework that integrates these technologies to support data collection and analysis in RD research.
DT4RD will implement a technology-driven platform designed to collect and analyse multimodal patient-generated data. The protocol includes three main components: (1) structured video capture to document clinical signs and functional performance, enabling remote assessment and computational analysis; (2) continuous physical activity monitoring using wearable sensors to quantify mobility and daily activity patterns and (3) digital questionnaires to capture patient-reported outcomes and contextual information. These data streams will be integrated within a dedicated digital infrastructure supporting secure data collection, storage and analysis. This pilot study will be conducted with participants affected by rare neuromuscular diseases to evaluate the feasibility, usability and analytical potential of the platform as well as its ability to generate clinically relevant insights from multimodal digital data anchored to regular hospital visits.
The study was approved in France by the Comité de Protection des Personnes Sud-Est III on 21 February 2023 (National Number ID-RCB 2022-A02749-34). UK approval was granted by the Health Research Authority and Health and Care Research Wales on 4 March 2024 (IRAS project ID 331474). The protocol has been registered on ClinicalTrials.gov under the number NCT05798325. All participants will be provided with verbal and written information about the study and its procedures before providing written informed consent. Data will be anonymised and handled in accordance with GDPR, French CNIL regulations (MR-001) and applicable national data-protection requirements. Results will be communicated through peer-reviewed publications, conference presentations and lay reports.
Recognising and acting on the connection to Country as a determinant of Indigenous peoples’ well-being is necessary to improve health inequities. Indigenous Australians experience a greater burden of chronic liver disease and poorer outcomes due to ongoing impacts of colonisation across determinants of health. This is exacerbated by a gradient in health outcomes based on remoteness, lack of specialist healthcare services and barriers to access. Our research aims to explore better ways to provide chronic liver disease screening and surveillance for very remote Indigenous Australian communities using non-invasive technologies On-Country.
Using an innovative combination of Indigenous and Quantitative research methodologies, this project involves 11 communities across four very remote sites in South Australia and Western Australia. The study comprises three parts: (1) site engagement with Aboriginal health services and remote communities; (2) a 12-month liver check (screening) phase and (3) a 24-month liver monitoring (surveillance) phase. The liver monitoring phase will use a stepped-wedge randomised controlled trial design where sites will have usual hepatocellular carcinoma (HCC) monitoring for a period of between 6 and 18 months and then On-Country monitoring for a period of between 6 and 18 months depending on treatment-sequence allocation. Recommended HCC monitoring involves 6 monthly liver ultrasounds and serum alpha-fetoprotein as per the site’s usual care processes, where participants travel to regional centres for liver ultrasound. On-Country monitoring will involve liver ultrasound and serum tumour markers provided On-Country every 6 months. The primary outcome is the difference in adherence to surveillance On-Country compared with usual care. In addition to statistical and health economic methods, yarning circles have been incorporated to explore participant experiences, their knowledge of liver disease and views about the On-Country monitoring.
This study was granted ethics approval from the relevant national and state Aboriginal Health Research Ethics Committees. Findings will be reported to all participants and will be disseminated to the broader community and local health services. Translation of outcomes will be supported by key Indigenous Australian and healthcare stakeholders, including peak health bodies and consumer groups. Dissemination with the academic community will be through peer-reviewed publications and presentations at relevant conferences.
ACTRN12625000256471.
Equity, diversity and inclusion (EDI), patient engagement and shared decision-making are important considerations throughout clinical trials, including the research ethics review stage. Meaningfully integrating these considerations can enhance the relevance and generalisability of trial results and reduce participation barriers among equity-deserving populations. Presently, it is unclear to what extent such guidance is provided at the ethics application stage for clinical trials. This study aimed to report the degree of guidance on EDI, patient engagement and shared decision-making in clinical trial research ethics documents.
This was an embedded mixed methods study conducted in collaboration with Clinical Trials Ontario.
This study analysed research ethics board (REB) forms and templates from 17 institutions across seven provinces in Canada.
15 REB application forms, 9 protocol templates and 17 informed consent document (ICD) templates were assessed for guidance related to EDI, patient engagement and shared decision-making. The Place of residence, Race, ethnicity, culture and language, Occupation, Gender and sex, Religion, Education, Socio-economic status, Social capital (PROGRESS)-Plus framework, International Association for Public Participation Spectrum of Public Participation, Patient-Oriented Research Level of Engagement Tool, Indigenous Research Level of Engagement Tool and shared decision-making standards guided our coding. We engaged with patients and persons with lived experience to inform interpretation, reporting and dissemination.
EDI guidance from 15 ethics application forms and 9 protocol templates predominantly covered the ‘Race, ethnicity, culture, language’ (n=14; 93.3%), ‘Age’ (n=13; 86.7%) and ‘Gender and sex’ (n=12; 80%) categories of PROGRESS-Plus but lacked nuance on diverse gender identities (n=1; 6.7%). Patient engagement guidance mostly covered the ‘inform’ level (n=7; 46.7%) and applying ‘knowledge in practice’ with non-Indigenous (n=7; 46.7%) or Indigenous communities (n=13; 86.7%). All 17 (100%) ICD templates included guidance on information about options, disclosures, key elements, ethical issues and study design. No guidance was available on time-dependent relationships, empowering patients and communities in co-leading trials or providing structured guidance in making trial participation decisions (all n=0; 0%).
We provided a comprehensive view of EDI, patient engagement and shared decision-making guidance in trial ethics applications in Canada. REB guidance may be strengthened in several areas to support the inclusion of equity-deserving populations in trials, meaningful engagement with patients and Indigenous communities and evidence-informed, values-aligned decisions about trial participation.
To evaluate the quality of action research studies using the Quality Assessment Action Research Checklist (QuARC) and to assess its utility as a tool for quality appraisal.
A hybrid systematic narrative review following Turnbull et al.'s six-stage methodology and reported in accordance with PRISMA 2020 guidance.
Scopus was searched for author self-identified action research studies published between January 2020 and March 2024.
Two reviewers independently selected studies meeting inclusion criteria: health science action research papers addressing any or all of QuARC's four quality factors. A scoring system was used to capture each of QuARC's 17 quality items, which was scored as 0 (absent), 0.5 (partial) or 1 (comprehensive). Narrative synthesis was undertaken across the four QuARC domains.
Thirty-two studies met the inclusion criteria. Reporting frequencies across QuARC were: Context (92.5%, mean = 3.7/4), Quality of Relationships (55% mean = 2.2/4), Quality of Action Research Process (62.5% mean = 2.5/4), and Quality of Outcomes (62.5% mean = 3.1/5). Reporting gaps were most evident in reflexive co-analysis, relational evaluation and explicit theoretical contribution.
Global reporting of rigour and quality in action research remains inconsistent. QuARC functioned both as an appraisal instrument and as an analytic lens, revealing systematic patterns in how action research privileges practical change over theoretical articulation and reflexive relational work. Further refinement and validation are recommended to strengthen its reliability as an appraisal tool.
Findings highlighted a critical need to establish a standardised, validated approach to assess quality in action research. Adoption of QuARC can enhance consistency, clarity and comparability across studies, strengthening the evidence base for action research methodologies.
This first systematic synthesis of QuARC's application provides an evidence base for its further development. This lays foundations for international standards in quality appraisal, strengthening the credibility, reproducibility and influence of action research.
To explore the views of community registered general nurses and directors of public health nursing on the current and future role of the community registered general nurse in the Republic of Ireland.
Anonymous cross-sectional descriptive survey.
Two questionnaires were developed; one targeted at community registered general nurses and one targeted at assistant directors of public health nursing or directors of public health nursing who were working with community registered general nurses. Social media was used to recruit participants. Descriptive statistics were used while data from open-ended questions were analysed using NVivo software.
A total of 97 community registered general nurses and 28 assistant directors of public health nursing or directors of public health nursing completed the surveys in 2023. There was consensus that community registered general nurses provide holistic care, including case management of adults with complex health needs living in the community. However, lack of promotional opportunities coupled with poor remuneration has resulted in job dissatisfaction. Respondents felt that community registered general nurses should focus on older adults, whereas public health nurses should focus on child health.
The role of the community registered general nurse needs to be clarified, and a promotional pathway developed to attract new graduates to this post.
This paper outlined the current role and vision for the future role of community registered general nurses.
CROSS guidelines.
No patient or public contribution.
This paper contributes to the challenges community nurses face regarding increased demand for community nursing, lack of career structure for some community nurses, and difficulties with staff retention within the community.
by Seung-Schik Yoo, Anvita Reddy, William Carroll, Kanyapat Ploypradith
Pharmacological removal of amyloid beta protofibrils has emerged as a promising therapeutic strategy to delay the onset of Alzheimer’s disease (AD) symptoms. As a non-pharmacological and noninvasive alternative, transcranial application of low-intensity ultrasound through intact skull can induce convective acoustic streaming, which has been shown to enhance cerebrospinal fluid solute transport and facilitate the clearance of interstitial solutes. This has led to the development of device-based approaches aimed at removing the precursors of amyloid beta (Aβ) plaques and mitigating cognitive decline in AD. We applied non-thermal, non-cavitational ultrasound (400 kHz frequency) in a pulsed mode (75 ms pulse duration, 2 Hz repetition rate) to the hippocampal region of male 5xFAD mice for 30 minutes weekly, starting at 10 weeks of age and continuing for 15 weeks (until 6 months of age). Spatial and recognition memory performance was assessed monthly using the Y-maze spontaneous alternation (SA) and novel object recognition (NOR) tests. A control group of age-matched mice underwent the same procedures with receiving zero acoustic output. Mice subjected to transcranial ultrasound (tUS) treatment maintained both SA and NOR performance throughout the entire experimental period, whereas mice that received sham tUS exhibited a progressive decline in memory beginning at 3–4 months of age. Congo Red staining of the brain sections revealed a significant (> 40%) reduction in Aβ plaques in the sonicated group. Histological analysis confirmed that repeated ultrasound exposure did not cause any detectable tissue damage. These findings suggest that low intensity tUS may serve as a novel, noninvasive therapeutic strategy to delay the onset of AD symptoms through the reduction of Aβ burden.
The aim of this scoping review was to identify factors associated with delayed initiation of end-of-life care for adult patients with cardiovascular disease in critical care settings.
Scoping review.
This scoping review was designed according to the Joanna Briggs Institute methodology. Included articles were uploaded and examined in Covidence.
A systematic search of bibliographic databases (CINAHL, Medline and Embase) and Google Scholar was performed to identify relevant literature between January 2003 and April 2025. Key search concepts included ‘end of life’, ‘cardiac’ and ‘critical care’.
A total of 9430 articles were initially identified. After removing 7750 irrelevant articles, 207 full-text articles were assessed for eligibility. A total of 34 articles were included in the final review. Four major themes were identified: (1) confidence in communication regarding end of life; (2) transition from active therapies to end-of-life care; (3) role clarity in the initiation and provision of end-of-life care; and (4) breakdown in the shared decision-making process.
Delayed initiation of end-of-life care for patients with cardiovascular disease in critical care settings could be mitigated through training to improve confidence when discussing end of life with patients and families, and utilisation of prognostic prediction assessment tools. Increased focus on inter-professional collaboration and shared decision-making in family meetings may reduce indecision at end of life.
The results were reported using the Preferred Reporting Items for Systematic Reviews and Meta-Analyses extension for Scoping Reviews.
This study did not include patient or public involvement in its design, conduct or reporting.
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